Intercept Pharmaceuticals Inc (NASDAQ:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced the results of a 72-week Phase 2 dose ranging trial of obeticholic acid (OCA), Intercept’s lead FXR agonist, in adult patients with nonalcoholic steatohepatitis (NASH) in Japan. The trial was conducted by Intercept’s collaborator, Sumitomo Dainippon Pharma.

This trial is the first to evaluate the safety and efficacy of OCA in Japanese NASH patients. The primary efficacy analysis was conducted on an intention to treat (ITT) basis, testing the dose dependent effects of once daily OCA (10mg, 20mg and 40mg) versus placebo on the primary endpoint of a two point improvement in the NAFLD Activity Score (NAS) with no worsening of fibrosis. The ITT analysis included all randomized patients who received treatment (50 per group), and patients who discontinued or did not have a repeat biopsy were treated as non-responders. A pre-specified completer analysis was conducted on the patients who had biopsies at both baseline and 72 weeks (45, 44, 44 and 37 patients in the placebo, 10mg, 20mg and 40mg OCA groups, respectively).

The ITT results in the table below show a dose dependent increase in the percentage of OCA treated patients compared to placebo who achieved the primary endpoint (p=0.053, not significant). The 40mg OCA dose group achieved statistical significance on the primary endpoint compared to placebo (p=0.0496). Dose-dependent trends not reaching statistical significance were also observed for several other pre-specified histologic endpoints, including the proportion of patients with steatosis and inflammation improvement, ballooning resolution and NASH resolution. No difference was seen in fibrosis improvement in the OCA groups compared to placebo.

placebo

10mg

20mg

40mg

ITT results

N=50

N=50

N=50

N=50

NAS improvement ≥2 pointswith no worsening of fibrosis

10 (20%)

11 (22%)p=0.8070**

14 (28%) p=0.3378**

19 (38%)p=0.0496**

p=0.053*

* Primary efficacy analysis is a stratified Cochran-Armitage test with multiple contrast coefficients. Statistical significance is based on a p-value < 0.05.

** The secondary efficacy analysis is a CMH (Cochran-Mantel-Haenszel) test stratified by baseline fibrosis stage for pairwise comparison of each OCA group vs. placebo group. The multiplicity was not adjusted.

In the completer analysis, similar dose dependent effects were observed, with 51% of patients in the 40mg dose group compared to 22% in the placebo group meeting the primary endpoint (p=0.0061).

With the exception of dose dependent pruritus, OCA appeared to be generally safe and well tolerated. The number of pruritus associated discontinuations were 0, 0, 2 and 5 patients in the placebo, 10mg, 20mg and 40mg OCA groups, respectively. Changes in lipid parameters, including LDL-C, HDL-C and triglycerides, appeared to be consistent with previously reported lipid changes in Western NASH patients. No other meaningful differences in the rate of adverse events between the OCA and placebo groups were noted.

“This study provides the first data on safety, efficacy and dose effects for OCA in Japanese NASH patients and we look forward to continuing to work with our partner Sumitomo Dainippon Pharma to understand the results further,” said Mark Pruzanski, MD, Chief Executive Officer and President of Intercept. “There were distinct differences in baseline characteristics in this study population when compared to the Western patients in the previously conducted FLINT trial. We are currently actively enrolling our Phase 3 REGENERATE trial, which was designed based on the robust FLINT results to evaluate OCA’s safety and efficacy in a similar Western patient population.” (Original Source)

Shares of Intercept Pharmaceuticals closed yesterday at $167.64. ICPT has a 1-year high of $314.88 and a 1-year low of $128.50. The stock’s 50-day moving average is $178.34 and its 200-day moving average is $232.03.

On the ratings front, Intercept has been the subject of a number of recent research reports. In a report issued on August 31, Wedbush analyst Liana Moussatos maintained a Buy rating on ICPT, with a price target of $493, which represents a potential upside of 194.1% from where the stock is currently trading. Separately, on August 13, Morgan Stanley’s Andrew Berens initiated coverage with a Sell rating on the stock and has a price target of $165.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Liana Moussatos and Andrew Berens have a total average return of 13.2% and 11.2% respectively. Moussatos has a success rate of 38.5% and is ranked #211 out of 3804 analysts, while Berens has a success rate of 55.6% and is ranked #993.

Overall, one research analyst has rated the stock with a Sell rating, 3 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $392.50 which is 134.1% above where the stock closed yesterday.

Intercept Pharmaceuticals Inc is a biopharmaceutical company. The Company is engaged in the development and commercialization of novel therapeutics to treat chronic liver disease utilizing its proprietary bile acid chemistry.